Translational Neuroscience

Our Research

The brain and spinal cord together form the nervous control centre of our body. This is where impulses originate that are transmitted to all muscles and other organs via the nervous system. Disorders of the brain-nerve-muscle axis are common, difficult to diagnose, disabling, often undertreated and accordingly, a severe burden for the individual and society.

Multidisciplinary translational research

Within the Medical Research Profile 'Translational Neuroscience', innovative multidisciplinary research is performed on a number of severe disorders of the brain, nerves and muscles. Clinicians and researchers work closely together on key clinical questions to unravel the molecular basis and identify novel intervention targets of the disease.

Our mission is to promote Translational Neuroscience research and education at LUMC to the benefit of patients and society

Prof. Michel Ferrari, chair of the medical research profile

Translational Neuroscience is one of the few medical research centres in the Netherlands with a very strong patient-oriented (translational) neuroscientific research focus. Many of our research themes are recognized national referral centres and the clinicians and researchers are national and international leaders in their field.

Focus on two major themes

Our research is divided in two complementary lines.

Paroxysmal Cerebral Disorders

NeurogeneticsThis research line primarily targets the cause, diagnosis and treatment of a number of common brain disorders with a significant impact on patients and society. The most important diseases are: migraine, epilepsy, sleep disorders, syncope, chronic pain and depression. Interestingly, these apparently distinct disorders have strong commonalities; many patients have multiple paroxysmal cerebral disorders, the attacks tend to become chronic on medication and the diseases are often caused by poorly functioning communication between nerve cells.  Consequently, certain brain regions become hypersensitive to specific stimuli, leading to disease attacks.

Central research questions include: What triggers attacks and how can we predict and prevent them? Why do symptoms intensify in some patients leading to a chronic condition, and how can we treat or prevent this? Which biomarkers may help improve diagnosis, prognosis and response to therapy, and help us understand disease mechanisms?

Neurological Motor Disorders

Biopsy DMD MuscleOur research also targets the cause, course and treatment of a number of congenital and acquired muscle and neurodegenerative diseases that impair normal movement. These include diseases like Duchenne and Facioscapulohumeral muscular dystrophy characterized by muscle weakness and wasting (atrophy) and myasthenia, caused by a defect in the transmission of nerve impulses to muscles. Additionally, research focuses on disorders such as Huntington and Parkinson disease, with progressive movement impairments caused by a dysfunction of the central brain control.

In all these diseases, patients experience impaired control of their muscles, which leads to severe disability and not infrequently premature death. Research aims at understanding disease mechanisms, natural history and developing new treatment methods.

Multidisciplinary integration of research

The two main research lines are based on disease themes. Interestingly, there are several important biological and molecular phenomena connecting the two research lines. To facilitate multidisciplinary crosstalk, we have established the following Task Forces:


Many departments within our Medical Research Profile perform research in the pharmacology arena, either by identifying novel drug targets or conducting clinical trials with "conventional" or "biological" drugs. Multidisciplinary work discussions efficiently facilitate optimal designs.

Omics and Imaging

Understanding disease mechanisms demands understanding the molecular, functional and anatomical networks. Neurological disorders are particularly dependent on non-/ minimally invasive techniques to obtain reliable read outs for diagnostic, prognostic and intervention monitoring purposes. To this end, Translational Neuroscience has established pipelines for various imaging modalities, sequencing, transcriptomics, proteomics, and metabolomics. For the Omics technologies, blood and cerebrospinal fluid (CSF) are the prime resources. The Biostatistics / Bioinformatics support is instrumental to the analysis of big-data.

Regenerative and functional neurobiology

CellsTo support the translational pipeline, a variety of disease models are instrumental to functional and intervention research. Our research groups have developed cutting edge models for neuronal and muscle (patho)biology. To further support the in vitro and in vivo model work, we have established an iPS platform in close collaboration with the central LUMC iPS facility. Ongoing studies are aiming to further develop this platform towards 3D tissue modelling (Brain, Muscle & Neuromuscular Junction-on-chips).

Synapse biology

The synapse is the central functional structure – node - in neuron/neuron and neuron/muscle communication. Many of our research lines converge at this structure. The function of the synapse can be modulated in various ways: genetics, autoimmunity, pharmacology, etc.


Leiden University Research Profile

Leiden Center for Translational Neuroscience (LCTN) forms together with Leiden Institute for Brain and Cognition (LIBC) the University Profile Brain Function and Dysfunction over the Life Span. This consortium provides means to increase the collaboration and crosstalk between various important neuroscientific fields like Medicine, Psychology, Linguistics, Social Sciences, etc.

Research Networks

BiobankingOur departments and groups perform cutting edge multidisciplinary biomedical research and collaborate closely with numerous academics and non-academic groups and consortia in the Netherlands and abroad.


Our Neuroscience research is instrumentally supported by many of the LUMC core facilities, in particular: Leiden Genome Technology Centre (LGTC), Biomedical Imaging, Center for Proteomics and Metabolomics, Animal Facility, Transgenesis Facility,  iPS Facility, iPS hotel, and Medical Statistics and Bioinformatics.

Dutch science agenda

The medical research profile Translational Neuroscience participates in the Nationale Wetenschapsagenda (in Dutch).